Discovery of RRx-001, a Myc and CD47 Downregulating Small Molecule with Tumor Targeted Cytotoxicity and Healthy Tissue Cytoprotective Properties in Clinical Development

After extensive screening
of aerospace compounds in an effort to
source a novel anticancer agent, RRx-001, a first-in-class dinitroazetidine
small molecule, was selected for advancement into preclinical and
clinical development. RRx-001 is a minimally toxic small molecule
with a distinct chemical structure and mechanism of action. The paradox
of RRx-001 is that it mediates both antitumor cytotoxicity and normal
tissue protection. The question of exactly how RRx-001 does this,
and by means of what mechanism(s), depending on the route of delivery,
intravenous or intratumoral, are explored. RRx-001 is currently in
phase 2 and 3 clinical trials for the treatment of multiple solid
tumor malignancies and as a supportive care drug.